First-approved gene therapy Glybera not a winner for uniQure despite $1M price and no competition
May 4 2016, 11:22 ET | About: uniQure N.V. (QURE) | By: Douglas W. House, SA News Editor Contact this editor with comments or a news tip
Some investors were expecting big things out of uniQure (QURE -11.4%) when the European Commission approved its gene therapy, Glybera (alipogene tiparvovec), in November 2012 for the treatment of the ultra-rare condition known as lipoprotein lipase deficiency. Patients with the inherited disorder lack an enzyme required to process fat. It affects only about 200 Europeans so the company priced the therapy at $1M per regimen, making it the most expensive drug in the world and an expected growth driver for the company.Since approval, however, Glybera has been commercially used in but one patient. Reimbursement has only been established in Germany and even there patient access is daunting. A recent example from a Berlin physician is informative. She had to complete a thesis-size application for regulators and personally call the CEO of an insurer to ask him to pay the $1M price tag.The marketing investment required to ramp Glybera was apparently too daunting for uniQure. It dropped plans for U.S. approval and sold the European rights to Italian drug maker Chiesi Farmaceutici, a small outfit with scant experience selling such a complex therapy.QURE has lost ~67% of its value since peaking at ~$36 in late September 2015.